What Happened? The healthcare company Grifols announced in a recent press release that it has enrolled and dosed the very first patient in a new Phase 3 clinical trial called…
Sanofi has announced encouraging results from a Phase 2 clinical trial of efdoralprin alfa, an investigational therapy for alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder affecting the liver and…
The Rady Children’s Institute for Genomic Medicine (RCIGM) has announced a landmark international collaboration with Sidra Medicine in Qatar to launch the BeginNGS® newborn genome sequencing program—marking a significant step…
The Alpha-1 Foundation (A1F) has announced a groundbreaking advancement in the fight against Alpha-1 Antitrypsin Deficiency (AATD) with the launch of AlphaDetect, a dedicated non-profit subsidiary focused on revolutionizing the…
Clinical studies are critical tools in identifying and developing therapeutics for a wide variety of conditions. This research is especially important in the rare disease and chronic illness spaces,…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
According to a story from globenewswire.com, the biopharmaceutical company Mereo BioPharma Group plc has recently announced that the company's investigational therapeutic alvelestat has earned Fast Track designation from the US…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Ryner Lai, MBBS, a medical graduate of Queen Mary, London University, is a physician and writer with a passion to use medical research as a way of improving patients’…
Takeda and its collaborator, Arrowhead Pharmaceuticals, recently announced results from AROAAT-2002 of the investigational drug fazirsiran. The drug is an RNA interference therapeutic developed to lessen alpha-1 antitirypsin protein…
Discerna Pharmaceuticals has just announced the results from a Phase 1 trial investigating their GalXC™ RNAi therapy (Belcesiran) for alpha-1 antitrypsin (AAT) deficiency-associated liver disease (AATLD). The results demonstrated both…
Arrowhead Pharmaceuticals recently announced positive twenty-four-week biopsy results from four patients who participated in the first cohort of the Phase II clinical trial of ARO-AAT. ARO-AAT is Arrowhead’s investigational…
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